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This pilot phase II trial studies how well ponatinib hydrochloride works in treating patients
with biliary cancer that has spread to other places in the body and that have alterations
(fusions) in a gene known as fibroblast growth factor receptor 2 (FGFR2). Ponatinib
hydrochloride may stop the growth of tumor cells by blocking some of the enzymes needed for
cell growth.

I. Establish preliminary correlations between FGFR2 fusions and evidence of any clinical
benefit.

II. Assess preliminary evaluation of FGFR2 pathway perturbation with ponatinib. III. To
describe patient-reported health-related quality of life and symptoms.

OUTLINE:

Patients receive ponatinib hydrochloride orally (PO) once daily (QD) on days 1-28. Courses
repeat every 28 days in the absence of disease progression or unacceptable toxicity.

After completion of study treatment, patients are followed up every 6 months for at least 2
years.

Inclusion Criteria:

- Histological/cytological confirmation of biliary cancer

- Confirmation of advanced biliary cancer that is refractory or intolerant to
gemcitabine or fluoropyrimidine based therapy with FGFR2 fusion [using next-gen
sequencing assays (such as Foundation One) or fluorescent in situ hybridization (FISH)
break-apart assays] or FGFR pathway mutation/amplification [using next-gen sequencing
assays (such as Foundation One)]; assays must be performed in a Clinical Laboratory
Improvement Amendments [CLIA] certified laboratory and done as a CLIA validated test
or research use only [RUO] in a CLIA laboratory

- Willing to return to enrolling institution for follow-up (during the Active Monitoring
Phase of the study); Note: during the Active Monitoring Phase of a study (i.e., active
treatment and observation), participants must be willing to return to the consenting
institution for follow-up

- Female and male patients who are fertile agree to use an effective form of
contraception with their sexual partners from registration through 4 months after the
end of treatment

- Ability to complete questionnaire(s) by themselves or with assistance

Exclusion Criteria:

- Any of the following:

- Pregnant women

- Nursing women

- Men or women of childbearing potential who are unwilling to employ adequate
contraception

- Co-morbid systemic illnesses or other severe concurrent disease which, in the judgment
of the investigator, would make the patient inappropriate for entry into this study or
interfere significantly with the proper assessment of safety and toxicity of the
prescribed regimens

- Immunocompromised patients and patients known to be human immunodeficiency virus (HIV)
positive and currently receiving antiretroviral therapy; NOTE: patients with a known
history of HIV infection are not eligible for this trial

- Previous or concurrent malignancy except adequately treated basal or squamous cell
skin cancer, in situ carcinoma of the cervix, or other solid tumor treated curatively
and without evidence of recurrence for at least 5 years

- Congestive heart failure within 6 months prior to registration, or left
ventricular ejection fraction (LVEF) less than lower limit of normal per local
institutional standards within 6 months prior to registration

- History of clinically significant (as determined by the treating physician)
atrial arrhythmia

- Any history of ventricular arrhythmia

- Active venous thromboembolism including deep venous thrombosis or pulmonary
embolism that is not amenable to treatment with anticoagulants